Orphan Drug Designation for ALS Treatment, Virtual Assistants in Genetic Counseling, Clinical Research Expansion in Asia

March 30, 2022 | New orphan drug designation for ALS treatment, virtual assistants in genetic counseling, AI in healthcare professional engagement, AI-powered pathology for clinical diagnostics, clinical research expansion in Asia, more.

eClinicalHealth, in collaboration with the Department of Dermatology at Wake Forest University School of Medicine, has created a digitized version of the SAPASI instrument, allowing researchers to monitor and record moderate-to-severe plaque psoriasis in clinical trial patients. A part of eClinicalHealth’s growing decentralized clinical trial (DCT) research platform known as Clinpal, this new SAPASI ePRO tool prompts patients on any device including their bring-your-own devices, at protocol-specified time intervals, to manually mark where their psoriasis is on a digital body map and answer several prompts pertaining to the severity of the psoriasis lesions. This data is then immediately compiled into a data model for researchers to use and monitor the effectiveness of the treatment. The digital map leverages Wake Forest’s validated diagnostic tool, the Self-administered Psoriasis Area Severity Index (SAPASI). Developed in 1995, the SAPASI was an upgrade from the Psoriasis Area Severity Index (PASI), which could only be evaluated by trained professionals who would observe the psoriasis in person. The modification of a self-administered version of the PASI allowed clinical studies to expand to more large-scale research of severe psoriasis. Press release.

Winterlight Labs will collaborate with leading memory clinic, Alzheimer Center Amsterdam of Amsterdam UMC, to decentralize assessments of patients’ cognition and function in the study of preclinical Alzheimer’s disease. Funded by the Dutch Ministry of Health (Health~Holland), Alzheimer Center Amsterdam will develop a remote measurement toolbox, integrating Winterlight’s digital speech assessments to help capture subtle changes in cognitive health. It is anticipated that the work will establish a validated, fast, and frequent approach to monitoring patients in the earliest stages of Alzheimer’s disease, and ultimately help to provide detailed measurements of the impact of preventative treatments. The toolbox will integrate Winterlight’s machine learning-based technology that analyzes short snippets of speech to detect the extent of cognitive impairment associated with preclinical Alzheimer’s disease. Winterlight’s AI platform objectively measures speech and language patterns via hundreds of vocal biomarkers. The acoustics and content of recordings, collected from patients through their own devices, are assessed using natural language processing techniques to achieve objective and precise measurement of a patient’s speech patterns. Press release.

In support of Critical Path Institute’s (C-Path) mission to catalyze innovation that accelerates the path to a healthier world, C-Path is excited to announce the opening of its EU nonprofit headquarters in the Netherlands, which will focus on the development of existing and future activities in Europe. C-Path is recognized globally as a leader in regulatory science, with extensive knowledge and experience in seeking and obtaining regulatory endorsement of drug development tools (DDTs) or novel methodologies in cooperation with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA). All C-Path regulatory-endorsed tools and methodologies are freely available to the broader scientific community. The nonprofit’s headquarters in Amsterdam will extend and complement C-Path’s activities to improve public health, as well as share expertise, data, risks and costs to expedite advancements in global regulatory science by facilitating public-private partnerships with members from the biopharmaceutical industry, government regulatory agencies, academic institutions and patient groups in Europe. Press release.

InFlectis BioScience received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its investigational treatment for amyotrophic lateral sclerosis (ALS), IFB-088. In preclinical studies, IFB-088 has been shown to prolong the protective effect of the integrated stress response, giving cells additional time to repair or eliminate misfolded proteins that can result in diseases such as ALS. The company plans to initiate a Phase 2 trial in Europe (France and Italy) this summer. Press release.

OptraHEALTH in partnership with Gamidor Diagnostics (Israel) is starting a research project with Beilinson Hospital, part of Clalit Health, and Tel Aviv University. Beilinson Hospital will conduct a research study for its patients undergoing genetic counseling and education, an important aspect for patients to make informed decisions. As part of this study, Beilinson Hospital will send OptraHEALTH’s GeneFAX virtual assistants to patients and assess the quality, time, and scale of genetic counseling sessions using GeneFAX, a unique solution that enables any patient to conversationally obtain information. Gamidor Diagnostics is supporting the pilot in the region as the adoption of genetic testing services powered by technology is gaining wider adoption. Press release.

Thermo Fisher Scientific introduced a new large-volume electroporation system that allows cell therapy developers to more easily move from clinical development to commercial manufacturing. The Gibco CTS Xenon Electroporation System leverages a closed, highly flexible design to facilitate gene modifications without the use of traditional viral vectors. The electroporation approach enables the introduction of a payload into a cell by temporarily increasing cell permeability using an electrical pulse. Unlike existing large-volume cell therapy electroporation solutions on the market, the Gibco CTS Xenon Electroporation System features programmable, flexible electroporation conditions, offering cell therapy developers full control to optimize a variety of hard-to-transfect cell types and payloads. The system may be used as a standalone technology or integrated with the Gibco CTS Rotea Counterflow Centrifugation System as part of a modular, closed, and automated cell therapy manufacturing workflow. The CTS Xenon Electroporation System can transfect up to 2.5e9 cells in 25 mL in a sterile system, enabling rapid and efficient non-viral transfection for clinical manufacturing applications. In addition, it delivers up to 80% cell viability and 90% gene knockout transfection to seamlessly scale from the small-volume Neon Transfection System without the need for re-optimization of electroporation parameters. Press release.

IQVIA launched OCE+, the first of several new advancements to its leading life science customer engagement platform. OCE+ adds IQVIA’s Next Best recommendation engine—a daily workflow tool providing AI-driven recommendations for engaging healthcare professionals (HCPs)—to its Orchestrated Customer Engagement (OCE) platform, providing enhanced HCP experiences, improved productivity, and increased ROI. OCE+ uses IQVIA’s industry-leading data and advanced analytics to deliver AI-driven recommendations directly into the daily workflows of life sciences commercial teams. This provides commercial teams with HCP-specific actions to take, such as recommending the best time to engage and the best method of contact. Recommendations are delivered directly through the OCE platform, providing a simplified and seamless user experience. By enabling commercial teams with AI-powered personalization, decisions can be made with greater speed, accuracy, and confidence to improve the HCP experience and yield best results. Press release.

PathAI and Cleveland Clinic announced a five-year strategic collaboration that will focus on leveraging PathAI’s quantitative pathology algorithms both to conduct new translational research and for use as clinical diagnostics in multiple disease areas. This collaborative effort combines PathAI’s AI-based platforms with Cleveland Clinic’s clinical expertise and multi-modal data to unlock a broad implementation of next-generation pathology diagnostics. The collaboration will enable the digitization of hundreds of thousands of pathology specimens, creating millions of whole slide images across multiple disease areas. By linking this digital pathology data with clinical and molecular data, Cleveland Clinic and PathAI will have a rich multi-modal dataset to conduct research using PathAI’s technology platform and menu of pathology algorithms. The partnership also will provide educational opportunities for Cleveland Clinic faculty and trainees, with the goal of enabling the development and usage of AI-powered pathology diagnostics to improve patient care. Cleveland Clinic will become an equity holder in PathAI as part of this agreement. Press release.

Helio Health announced that Hepatology Communications published positive results from the ENCORE study validating HelioLiver's strong clinical performance in detecting the presence of early hepatocellular carcinoma (HCC), the most common form of liver cancer and the seventh most common cancer worldwide but the second in cancer-related mortality, often due to late diagnosis. In this prospective, blinded, multi-center Phase 2 study, HelioLiver demonstrated high specificity (91%) and high sensitivity (76%) in detecting early-stage (stage I and II) HCC, significantly outperforming other clinically available detection tools such as AFP (57%), GALAD (65%), and ultrasound (47%). When considering HCC overall, HelioLiver performed at an 85% sensitivity with the same 91% specificity. Helio developed its next-generation sequencing platform, ECLIPSE, to identify 77 methylation targets across 28 genes. These DNA markers work in combination with the serum HCC proteins AFP, AFP-L3%, and DCP to enhance the multi-analyte algorithm. Press release.

The Rare Genomics Institute (RG) is receiving support from Amazon Web Services (AWS) to enhance its RareShare website. RareShare is a unique social hub serving hundreds of rare disorder communities since 2008, allowing direct exchange of information among patients, families, research organizations, and healthcare professionals to improve the lives of those affected. AWS helps nonprofits—including those dedicated to health research and patient empowerment—use cloud technology to increase their impact. As part of the collaboration with the Rare Genomics Institute, AWS is providing unrestricted financial support funds for an array of RareShare projects, AWS computing credits, and project implementation support by dedicated AWS technical specialists. The Rare Genomics Institute plans to work with AWS to upgrade the RareShare website, modernizing the underlying software architecture, enhancing its appearance and navigational features, adding better cross-relational search capabilities, and expanding international access by adding language translation functions. With these improvements, current members will be able to more easily navigate through the website’s content, and new visitors will experience an appealing interface that encourages them to explore and become active RareShare members. Press release.

BC Platforms (BCP) has expanded its R&D operations in Singapore, which spearheads precision medicine activities in the Asia Pacific region, including China and Japan. The company also announced a research partnership with Singapore Health Services (SingHealth), Singapore’s largest public healthcare group. SingHealth has joined BCP’s BCRQUEST global data network in a research partnership to accelerate the genetic understanding and clinical research of diseases prevalent in Asian cohorts, including cancer and cardiovascular diseases. The core strategy of BCP is to develop privacy and security driven technology for trusted research infrastructure. This will then enable a safe build up cohort of studies representing Asia’s rich multi-ethnicity, which is of value for life science research as well as clinical decision making. The company’s Singapore R&D Centre will support recent Asia-based collaborations including with Japan’s Mitsubishi Space Software, announced Feb 2022, and Thailand’s Bumrungrad International Hospital (BIH), announced October 2021. Press release.

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